NCT01282697: RAPIRI

Clinical Trial of Rapamycin and Irinotecan in Pediatric Patients With Refractory Solid Tumors

Completed Phase 1 Last updated 22 December 2025

What this trial tests

Phase 1 trial testing Combined administration of irinotecan and rapamycin in Refractory Solid Tumors in Children in 42 participants. Completed in 20 February 2013.

Timeline

22 April 2011

Primary endpoint
20 February 2013

20 February 2013

Quick facts

Lead sponsor	University Hospital, Strasbourg, France
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	42
Start date	22 April 2011
Primary completion	20 February 2013
Estimated completion	20 February 2013
Sites	9 locations across France

Drugs / interventions tested

• Combined administration of irinotecan and rapamycin — full drug profile →

Conditions studied

• Refractory Solid Tumors in Children — all drugs for Refractory Solid Tumors in Children →

Sponsor

University Hospital, Strasbourg, France

Who can join

Adults 1 to 21, any sex, with Refractory Solid Tumors in Children. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Therapeutic solutions to treat solid tumors that are resistant to conventional treatments are now limited. Laboratory data in animals (on pediatric tumors such as brain tumors, sarcomas and neuroblastomas) have shown that the combination of irinotecan (HIF1alpha inhibitor) and rapamycin (mTOR inhibitor) allowed to block development of blood vessels in the tumor and could, in some cases, stop its progression. This drug combination has already been tested in adult patients with refractory tumors and seems to give encouraging results with stabilization of the tumor. The dose and toxicity of irinotecan and rapamycin are known when these drugs are administered separately and in a context different from that of refractory tumors. RAPIRI is a phase I clinical trial whose principal objectives are to determine the maximum dose at which these two molecules may be administered and to assess the safety of this new combination of drugs.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. The hypoxia-driven crosstalk between tumor and tumor-associated macrophages: mechanisms and clinical treatment strategies.
   Bai R, Li Y, Jian L, Yang Y, et al · · 2022 · cited 200× · PMID 36071472 · DOI 10.1186/s12943-022-01645-2
2. Advances in paediatric cancer treatment.
   Saletta F, Seng MS, Lau LM. · · 2014 · cited 50× · PMID 26835334 · DOI 10.3978/j.issn.2224-4336.2014.02.01
3. Regulation of Transactivation at C-TAD Domain of HIF-1<i>α</i> by Factor-Inhibiting HIF-1<i>α</i> (FIH-1): A Potential Target for Therapeutic Intervention in Cancer.
   Rani S, Roy S, Singh M, Kaithwas G. · · 2022 · cited 43× · PMID 35592530 · DOI 10.1155/2022/2407223
4. The hypoxia-inducible factor-1α in stemness and resistance to chemotherapy in gastric cancer: Future directions for therapeutic targeting.
   Ozcan G. · · 2023 · cited 21× · PMID 36846589 · DOI 10.3389/fcell.2023.1082057
5. Bone sarcomas: from biology to targeted therapies.
   Gaspar N, Di Giannatale A, Geoerger B, Redini F, et al · · 2012 · cited 21× · PMID 23226965 · DOI 10.1155/2012/301975
6. The therapeutic potential of targeting the PI3K pathway in pediatric brain tumors.
   Rogers HA, Estranero J, Gudka K, Grundy RG. · · 2017 · cited 13× · PMID 27926496 · DOI 10.18632/oncotarget.13781
7. Sirolimus Pharmacokinetics Variability Points to the Relevance of Therapeutic Drug Monitoring in Pediatric Oncology.
   Sabo AN, Jannier S, Becker G, Lessinger JM, et al · · 2021 · cited 10× · PMID 33808416 · DOI 10.3390/pharmaceutics13040470
8. Characterization of the transcriptional and metabolic responses of pediatric high grade gliomas to mTOR-HIF-1α axis inhibition.
   Nguyen A, Moussallieh FM, Mackay A, Cicek AE, et al · · 2017 · cited 10× · PMID 29069732 · DOI 10.18632/oncotarget.16500